Nucleic acids adapted for tissue specific expression

Industry

CPC
A61K48/0058

This industry / category may be too specific. Please go to a parent level for more data

Parent Industries

AHUMAN NECESSITIES A61Medical science A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases Gene therapy

Current Industry

A61K48/0058Nucleic acids adapted for tissue specific expression

Industries

Please log in for detailed analytics

Patents Grantslast 30 patents

Information Patent Grant
Pharmaceutical compositions for the treatment of cancer
- Patent number 12,310,936
- Issue date May 27, 2025
- Seleq Oy
- Mikael Björnstedt
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
AAV capsid variants and uses thereof
- Patent number 12,296,025
- Issue date May 13, 2025
- VOYAGER THERAPEUTICS, INC.
- Mathieu Emmanuel Nonnenmacher
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Transcription regulatory elements and uses thereof
- Patent number 12,296,026
- Issue date May 13, 2025
- Astellas Gene Therapies, Inc.
- John T. Gray
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized promoter sequences, intron-free expression constructs and...
- Patent number 12,290,574
- Issue date May 6, 2025
- SPARK THERAPEUTICS, INC.
- Xavier Anguela
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Heterologous targeting peptide grafted AAVs
- Patent number 12,291,719
- Issue date May 6, 2025
- UNIVERSITY OF MASSACHUSETTS
- Miguel Sena Esteves
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Genome editing methods and constructs
- Patent number 12,285,495
- Issue date Apr 29, 2025
- FONDAZIONE TELETHON ETS
- Alberto Auricchio
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Means and methods for AAV gene therapy in humans
- Patent number 12,285,451
- Issue date Apr 29, 2025
- Uniqure IP B.V.
- Bart Antonius Nijmeijer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus virions for treatment of epilepsy
- Patent number 12,263,231
- Issue date Apr 1, 2025
- Jichi Medical University
- Shin-ichi Muramatsu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Optimized mini-dystrophin genes and expression cassettes and their use
- Patent number 12,257,321
- Issue date Mar 25, 2025
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- A01 - AGRICULTURE FORESTRY ANIMAL HUSBANDRY HUNTING TRAPPING FISHING
Information Patent Grant
Optimised coding sequence and promoter
- Patent number 12,257,319
- Issue date Mar 25, 2025
- UCL BUSINESS LTD
- Amit Nathwani
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for NMNAT1-associated retinal degeneration
- Patent number 12,257,320
- Issue date Mar 25, 2025
- Massachusetts Eye and Ear Infirmary
- Eric A. Pierce
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Methods of treating non-arteritic anterior ischemic optic neuropathy
- Patent number 12,252,518
- Issue date Mar 18, 2025
- Life Biosciences, Inc.
- Michel Wathier
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Myosin 15 promoters and uses thereof
- Patent number 12,252,520
- Issue date Mar 18, 2025
- Decibel Therapeutics, Inc.
- Joseph Burns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Treatment of mucopolysaccharidosis IVA
- Patent number 12,247,213
- Issue date Mar 11, 2025
- The Nemours Foundation
- Shunji Tomatsu
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa
- Patent number 12,246,072
- Issue date Mar 11, 2025
- Stylianos Michalakis
- C07 - ORGANIC CHEMISTRY
Information Patent Grant
Modified ligand-gated ion channels and methods of use
- Patent number 12,247,064
- Issue date Mar 11, 2025
- Howard Hughes Medical Institute
- Scott Sternson
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
DNA vector for targeted gene therapy
- Patent number 12,246,073
- Issue date Mar 11, 2025
- GENETIC DIAGNOSTICS AND THERAPY 21 LTD
- Anton Gamolski
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus factor VIII vectors
- Patent number 12,239,692
- Issue date Mar 4, 2025
- Biomarin Pharmaceutical Inc.
- Peter Cameron Colosi
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Drug and gene therapy to treat high myopia and other ocular disorde...
- Patent number 12,233,050
- Issue date Feb 25, 2025
- City University of Hong Kong
- Wenjun Xiong
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic constructs for treating cancer
- Patent number 12,233,135
- Issue date Feb 25, 2025
- PRECISION MOLECULAR INC.
- Christopher Ullman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Myosin 15 promoters and uses thereof
- Patent number 12,233,136
- Issue date Feb 25, 2025
- Decibel Therapeutics, Inc.
- Joseph Burns
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
GLP-1 and use thereof in compositions for treating metabolic diseases
- Patent number 12,221,615
- Issue date Feb 11, 2025
- The Trustees of the University of Pennsylvania
- Christian Hinderer
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Therapeutic applications of CPF1-based genome editing
- Patent number 12,214,056
- Issue date Feb 4, 2025
- Duke University
- Charles A. Gersbach
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Optimized human clotting factor IX gene expression cassettes and th...
- Patent number 12,209,263
- Issue date Jan 28, 2025
- The University of North Carolina at Chapel Hill
- Xiao Xiao
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Messenger RNA therapy for the treatment of ornithine transcarbamyla...
- Patent number 12,201,677
- Issue date Jan 21, 2025
- Translate Bio, Inc.
- Frank DeRosa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Gene therapy for retinitis pigmentosa
- Patent number 12,201,698
- Issue date Jan 21, 2025
- Genzyme Corporation
- Catherine O'Riordan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Adeno-associated virus compositions for restoring HBB gene function...
- Patent number 12,201,657
- Issue date Jan 21, 2025
- City of Hope
- Saswati Chatterjee
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Compositions and processes for targeted delivery, expression and mo...
- Patent number 12,195,746
- Issue date Jan 14, 2025
- Combined Therapeutics, Inc.
- Romain Micol
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Grant
Materials and methods for treatment of hereditary haemochromatosis
- Patent number 12,186,406
- Issue date Jan 7, 2025
- CRISPR THERAPEUTICS AG
- Roman Lvovitch Bogorad
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Grant
Formulations for small intestinal delivery of RSV and norovirus ant...
- Patent number 12,186,407
- Issue date Jan 7, 2025
- Vaxart, Inc.
- George Trager
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Patents Applicationslast 30 patents

Information Patent Application
ADENO-ASSOCIATED VIRAL VECTOR COMPOSITIONS AND METHODS OF PROMOTING...
- Publication number 20250186619
- Publication date Jun 12, 2025
- NEW YORK UNIVERSITY
- Dounia ABBADI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITION FOR TREATING OR PREVENTING DISEASES, DISORDERS, OR COND...
- Publication number 20250186617
- Publication date Jun 12, 2025
- Restore Vision Inc.
- Toshihide Kurihara
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS FOR AND METHODS OF LONG-TERM INTEGRATION OF CIRCUITS U...
- Publication number 20250186620
- Publication date Jun 12, 2025
- DUKE UNIVERSITY
- Kafui Dzirasa
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
LENTIVIRAL VECTOR
- Publication number 20250188491
- Publication date Jun 12, 2025
- Genespire S.r.l.
- Elena Barbon
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
USING DREADD FOR NEURONAL MODULATION IN TREATING NEURONAL DISEASES
- Publication number 20250186610
- Publication date Jun 12, 2025
- Fadi ASSAF
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING GLYCOGEN ST...
- Publication number 20250186618
- Publication date Jun 12, 2025
- DUKE UNIVERSITY
- Baodong Sun
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
EXPRESSION SYSTEM FOR THE TREATMENT OF CANCER
- Publication number 20250186623
- Publication date Jun 12, 2025
- FUNDACION PARA LA INVESTIGACION MEDICA APLICADA
- Jesus María Prieto Valtueña
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
Molecular Systems and Therapies Using the Same
- Publication number 20250186616
- Publication date Jun 12, 2025
- Agency for Science, Technology and Research
- Choong Tat Keng
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS FOR NEURAL REGENERATION IN VIVO AND USES THEREOF
- Publication number 20250186622
- Publication date Jun 12, 2025
- The Board of Regents of the University of Texas System
- Yunjia Zhang
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MATERIALS AND METHODS FOR THE TREATMENT OF EIF2B5 MUTATIONS AND DIS...
- Publication number 20250186621
- Publication date Jun 12, 2025
- Research Institute at Nationwide Children's Hospital
- Kevin Flanigan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTION OF DUCHEN...
- Publication number 20250179450
- Publication date Jun 5, 2025
- The Board of Regents of the University of Texas System
- Yu ZHANG
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
COMPOSITION FOR USE IN THE TREATMENT OF FABRY DISEASE
- Publication number 20250177572
- Publication date Jun 5, 2025
- Takeda Pharmaceutical Company Limited
- Rizwana Islam
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SEQUENCE OF ACTIVATED PROTEIN C
- Publication number 20250177571
- Publication date Jun 5, 2025
- Jichi Medical University
- Tsukasa OHMORI
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
RECOMBINANT ADENO-ASSOCIATED VIRUSES FOR CNS TROPIC DELIVERY
- Publication number 20250179518
- Publication date Jun 5, 2025
- Regenxbio Inc.
- Olivier Danos
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MULTIPLEXED REPRESSION OF IMMUNOSUPPRESSIVE GENES
- Publication number 20250177570
- Publication date Jun 5, 2025
- Yale University
- Sidi Chen
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
METHODS AND MEANS FOR THE PREVENTION AND/OR TREATMENT OF HEMOPHILIC...
- Publication number 20250170275
- Publication date May 29, 2025
- uniQure biopharma B.V.
- Ellen Broug-Holub
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...
- Publication number 20250161344
- Publication date May 22, 2025
- Avidity Biosciences, Inc.
- Arthur A. LEVIN
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE WITH...
- Publication number 20250163457
- Publication date May 22, 2025
- ASKLEPIOS BIOPHARMACEUTICAL, INC.
- Sam HOPKINS
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...
- Publication number 20250163458
- Publication date May 22, 2025
- Allen Institute
- Tanya Daigle
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT...
- Publication number 20250161490
- Publication date May 22, 2025
- Research Institute at Nationwide Children's Hospital
- Louise Rodino-Klapac
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR IN VIVO NUCLEASE-MEDIATED TREATMENT OF...
- Publication number 20250161493
- Publication date May 22, 2025
- The Trustees of the University of Pennsylvania
- Lili Wang
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SYNTHETIC CANCER-SPECIFIC PROMOTERS
- Publication number 20250161496
- Publication date May 22, 2025
- EARLI Inc.
- Dariusz WODZIAK
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
TRANSMEMBRANE RECEPTOR GENE EDITING
- Publication number 20250161494
- Publication date May 22, 2025
- Orthobio Therapeutics, Inc.
- Peter J. Millet
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
LIGAND GATED ION CHANNELS AND METHODS OF USE
- Publication number 20250163130
- Publication date May 22, 2025
- TRAMES BIO, INC.
- Anthony Lau
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
SFASL FOR INHIBITING RPE CELL DEATH AND ASSOCIATED DISORDERS
- Publication number 20250163131
- Publication date May 22, 2025
- ONL Therapeutics, Inc.
- Andrew KOCAB
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
BASE EDITING APPROACHES FOR THE TREATMENT OF BETA-THALASSEMIA
- Publication number 20250161492
- Publication date May 22, 2025
- INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
- Annarita MICCIO
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
METHODS OF GENERATING A POPULATION OF NEURONS FROM HUMAN GLIAL PROG...
- Publication number 20250161495
- Publication date May 22, 2025
- University of Rochester
- Steven A. Goldman
- A61 - MEDICAL OR VETERINARY SCIENCE HYGIENE
Information Patent Application
COMPOSITIONS AND METHODS FOR NEUROLOGICAL DISEASES
- Publication number 20250152742
- Publication date May 15, 2025
- TRAMES BIO, INC.
- Anthony Lau
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
MATERIALS AND METHODS FOR THE TREATMENT OF LIMB GIRDLE MUSCULAR DYS...
- Publication number 20250152740
- Publication date May 15, 2025
- Research Institute at Nationwide Children's Hospital
- Kevin Flanigan
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...
Information Patent Application
NUCLEIC ACID CONSTRUCTS ENCODING A CELL PENETRATING PEPTIDE, SIGNAL...
- Publication number 20250152741
- Publication date May 15, 2025
- London Health Sciences Centre Research Inc.
- Stephen Pasternak
- C12 - BIOCHEMISTRY BEER SPIRITS WINE VINEGAR MICROBIOLOGY ENZYMOLOGY MUTATION...

Nucleic acids adapted for tissue specific expression

Industry

Parent Industries

Current Industry

Patents Grantslast 30 patents

Pharmaceutical compositions for the treatment of cancer

AAV capsid variants and uses thereof

Transcription regulatory elements and uses thereof

Optimized promoter sequences, intron-free expression constructs and...

Heterologous targeting peptide grafted AAVs

Genome editing methods and constructs

Means and methods for AAV gene therapy in humans

Adeno-associated virus virions for treatment of epilepsy

Optimized mini-dystrophin genes and expression cassettes and their use

Optimised coding sequence and promoter

Gene therapy for NMNAT1-associated retinal degeneration

Methods of treating non-arteritic anterior ischemic optic neuropathy

Myosin 15 promoters and uses thereof

Treatment of mucopolysaccharidosis IVA

Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa

Modified ligand-gated ion channels and methods of use

DNA vector for targeted gene therapy

Adeno-associated virus factor VIII vectors

Drug and gene therapy to treat high myopia and other ocular disorde...

Therapeutic constructs for treating cancer

Myosin 15 promoters and uses thereof

GLP-1 and use thereof in compositions for treating metabolic diseases

Therapeutic applications of CPF1-based genome editing

Optimized human clotting factor IX gene expression cassettes and th...

Messenger RNA therapy for the treatment of ornithine transcarbamyla...

Gene therapy for retinitis pigmentosa

Adeno-associated virus compositions for restoring HBB gene function...

Compositions and processes for targeted delivery, expression and mo...

Materials and methods for treatment of hereditary haemochromatosis

Formulations for small intestinal delivery of RSV and norovirus ant...

Patents Applicationslast 30 patents

ADENO-ASSOCIATED VIRAL VECTOR COMPOSITIONS AND METHODS OF PROMOTING...

COMPOSITION FOR TREATING OR PREVENTING DISEASES, DISORDERS, OR COND...

COMPOSITIONS FOR AND METHODS OF LONG-TERM INTEGRATION OF CIRCUITS U...

LENTIVIRAL VECTOR

USING DREADD FOR NEURONAL MODULATION IN TREATING NEURONAL DISEASES

COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING GLYCOGEN ST...

EXPRESSION SYSTEM FOR THE TREATMENT OF CANCER

Molecular Systems and Therapies Using the Same

METHODS FOR NEURAL REGENERATION IN VIVO AND USES THEREOF

MATERIALS AND METHODS FOR THE TREATMENT OF EIF2B5 MUTATIONS AND DIS...

CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTION OF DUCHEN...

COMPOSITION FOR USE IN THE TREATMENT OF FABRY DISEASE

SEQUENCE OF ACTIVATED PROTEIN C

RECOMBINANT ADENO-ASSOCIATED VIRUSES FOR CNS TROPIC DELIVERY

MULTIPLEXED REPRESSION OF IMMUNOSUPPRESSIVE GENES

METHODS AND MEANS FOR THE PREVENTION AND/OR TREATMENT OF HEMOPHILIC...

NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON...

THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE WITH...

ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN...

ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT...

COMPOSITIONS AND METHODS FOR IN VIVO NUCLEASE-MEDIATED TREATMENT OF...

SYNTHETIC CANCER-SPECIFIC PROMOTERS

TRANSMEMBRANE RECEPTOR GENE EDITING

LIGAND GATED ION CHANNELS AND METHODS OF USE

SFASL FOR INHIBITING RPE CELL DEATH AND ASSOCIATED DISORDERS

BASE EDITING APPROACHES FOR THE TREATMENT OF BETA-THALASSEMIA

METHODS OF GENERATING A POPULATION OF NEURONS FROM HUMAN GLIAL PROG...

COMPOSITIONS AND METHODS FOR NEUROLOGICAL DISEASES

MATERIALS AND METHODS FOR THE TREATMENT OF LIMB GIRDLE MUSCULAR DYS...

NUCLEIC ACID CONSTRUCTS ENCODING A CELL PENETRATING PEPTIDE, SIGNAL...